A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme

Investigating the Safety of Adjusted Infusion Rates for an Investigational Medication

Not Recruiting
2 years - 65 years
All
Phase 4
8 participants needed

Study Overview

This Phase 4 study will evaluate the safety and tolerability of Fabrazyme at current approved dose with increases in the infusion rate and reduced infusion volume. This study aims to generate data to provide the guidance on how infusion rate can be safely increased and minimize the burden of the life-long treatment with Fabrazyme.

Study Details

The total duration will be up to 7 months

Eligibility Criteria

You may be eligible for this study if you meet the following criteria:

  • Conditions: Fabry's Disease
  • Age: 2 years - 65 years
  • Gender: All

Inclusion Criteria:

  • Participants with confirmed diagnosis of FD who are ≥2 and ≤65 years of age at the time of signing the informed consent form (ICF) or assent, if applicable.
  • Cohort 1: female participants with body weight ≥30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
  • Cohort 2: non-classic male participants with body weight ≥30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
  • Cohort 3: classic male participants with body weight ≥30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
  • Cohort 4: participants with body weight <30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.

Women of childbearing potential must use a highly effective method of contraception through the study.

Exclusion Criteria:

  • Female participants who are pregnant or breastfeeding.
  • History of significant allergic disease or hypersensitivity to Fabrazyme or other medicinal products.
  • Contraindication to Fabrazyme or any of the premedications or rescue medications (diphenhydramine, loratadine, cetirizine, fexofenadine, acetaminophen, montelukast, dexamethasone).
  • Any other medical condition considered to make the increased infusion rate not tolerable at the Investigator's discretion.

The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.

Updated on 28 Mar 2025. Study ID: NCT06019728

This study investigates the safety and tolerability of an investigational medication for individuals with Fabry Disease (FD). Fabry Disease is a rare genetic condition that affects the body's ability to break down a certain type of fat, leading to a buildup that can harm different parts of the body. The purpose of this study is to explore how increasing the infusion rate and reducing the infusion volume of the medication can be done safely.

Participants in the study will receive the investigational medication at an increased infusion rate. The study will monitor for any infusion-associated reactions (IARs), which are reactions that can happen when the medication is given into the vein. Participants will be grouped into different study arms based on their body weight and previous treatment history with the investigational medication.

  • Who can participate: Participants aged 2 to 65 years with a confirmed diagnosis of Fabry Disease may join. They must have been treated with the investigational medication for at least 3 months without any infusion-associated reactions.
  • Study details: Participants will receive the investigational medication at increased infusion rates. Infusion-associated reactions will be monitored.
  • Study Timelines: The study will last up to 7 months.